Targeting existing drug for rare bone cancer patients

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drug for rare bone cancer patients

Patients with a rare bone cancer of the skull and spine chordoma could be helped by existing drugs. Researchers suggest a clinical trial of PI3K inhibitors for this particular group of chordoma patients can benefit from existing treatments.

For the first time, scientists have used whole genome sequencing to gain a better understanding of the biology underlying chordoma. The results reveal promising new treatment options for a cancer with a poor prognosis.

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Drugs known as PI3K inhibitors

Scientists from the Wellcome Trust Sanger Institute, University College London Cancer Institute and the Royal National Orthopaedic Hospital NHS Trust. Scientists show that a group of chordoma patients have mutations in genes. Meanwhile, the target of existing drugs known as PI3K inhibitors.

 

The team studied chordoma tumours from 104 patients. Found that 16 per cent of the tumour samples had genetic changes, or mutations, in PI3K signaling genes. These genes are the target of existing drugs known as PI3K inhibitors. Inhibitors to treat cancer, including breast cancer, lung cancer and lymphoma, but have not yet been considered for chordoma.

Chordoma is a rare form of bone cancer that strikes individuals of all ages. Chordomas are slow-growing aggressive and life-threatening tumours. Tumour form in the vertebral bodies of the spine, the sacrum and base of the skull. The cancerous tumour considered to develop from persistent embryonic tissue known as the notochord. Further, as they involve critical structures such as the brainstem, spinal cord, and important nerves and arteries, chordomas are difficult to treat. Surgery and radiation are currently the only effective forms of treatment.

Dr Sam Behjati, from the Wellcome Trust Sanger Institute, said “By sequencing the tumours’ DNA, we get a much clearer view of the genetic changes that drive chordoma. However, We have shown that a particular group of chordoma patients treated with PI3K inhibitors, based on their mutations.”

New cancer gene specific to chordoma known as LYST

Moreover, the gene brachyury or T known for some time to play a role in chordoma. But for first time the team use genomic sequencing. To demonstrate that only one additional copy of the T gene drives these tumours. The team also discovered new cancer gene specific to chordoma known as LYST not found in any other cancer.

Josh Sommer, a survivor of chordoma and Executive Director of the Chordoma Foundation, USA, said “These findings represent a major step forward in understanding the underlying causes of chordoma, and provide hope that better treatments may soon be available for some patients.”

Professor Adrienne Flanagan, joint lead author from the Royal National Orthopaedic Hospital NHS Trust and University College London Cancer Institute, said “This study provides a resource for chordoma research for years to come. In the future we hope to target chordoma from three angles trialling PI3K inhibitors in chordoma.

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Although, developing a therapeutic approach to switching off the extra copy of the T gene and studying the function of LYST as a cancer gene and its role in the development of chordoma”.

 

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